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Full Prescribing Information (PDF)

Mucopolysaccharidosis I (MPS I) is a rare, autosomal recessive genetic disease that affects multiple organ systems and tissues. The disease is caused by a defect in the gene coding for the lysosomal enzyme alpha-L-iduronidase. As a result of this defect, the cells of people with MPS I are either unable to produce the enzyme or produce it in low amounts.

This results in an inability of the lysosome to act in the stepwise degradation of certain glycosaminoglycans (GAG) -- namely dermatan sulfate and heparan sulfate. This process is essential for normal growth and homeostasis of tissues.[1],[2] These glycosaminoglycans, which are important constituents of the extra cellular matrix, joint fluid, and connective tissue throughout the body, progressively accumulate in the lysosome. Ultimately the accumulation causes cell, tissue, and organ dysfunction by largely unknown pathophysiological mechanisms.

MPS I is considered to be the prototypical lysosomal storage disorder with progressive multi-systemic disease and presenting features that vary depending on where a patient is on the disease continuum.


Clinical manifestations of MPS I show a chronic and progressive course that is multisystemic in nature. The disease is highly heterogeneous, spanning a spectrum of severity. Individuals with the most severe form of MPS I typically suffer from a number of progressively debilitating symptoms, including mental retardation. [2] Their life span is less than 10 years. [3] Individuals at the less severe end of the disease spectrum can have some of the same physical symptoms, but they generally retain normal intellect and stature, and may have a normal life span. Hurler, Hurler-Scheie, and Scheie syndromes are historical terms used to describe the most severe, intermediate, and least severe forms of MPS I. However, because there is no clear delineation between these classifications, patients are best described as having severe or less severe MPS I disease.[2]

For more information on MPSI, download our slide set entitled

A Clinical Profile of MPS I symptoms, diagnosis, and medical management. (PDF)


References:
1. Clarke LA. Clinical diagnosis of lysosomal storage diseases. In: Organelle Diseases. Clinical Features, Diagnosis, Pathogenesis and Management. Applegarth DA, Dimmick JE, Hall JG, editors. London: Chapman and Hall Medical; 1997. p. 45-8.

2. Neufeld EF, Muenzer J. The mucopolysaccharidoses. In: Scriver CR, Beaudet AL, Sly WS, Valle D, Childs B, Kinzler KW, et al, editors. The Metabolic and Molecular Bases of Inherited Disease. Vol. III. 8th ed. New York: McGraw-Hill; 2001. p. 3421-8.

3. Cleary MA, Wraith JE. The presenting features of mucopolysaccharidosis type IH (Hurler syndrome). Acta Paediatr. 1995;84:337–339.


Region/Country
This site is intended for use in the United States. Please visit the Genzyme site for your country or region.
Even though people with MPS I have the same enzyme deficiency, they may experience a wide range of symptoms.
Information on MPS I disease is limited because of its rarity. Learn more about MPS I Registry, a global resource dedicated to improving the understanding of MPS I disease.

ALDURAZYME® (laronidase) is indicated for patients with Hurler and Hurler-Scheie forms of Mucopolysaccharidosis I (MPS I) and for patients with the Scheie form who have moderate to severe symptoms.  The risks and benefits of treating mildly affected patients with the Scheie form have not been established.

 

ALDURAZYME has been shown to improve pulmonary function and walking capacity.  ALDURAZYME has not been evaluated for effects on the central nervous system manifestations of the disorder.

 

Important Safety Information

 

WARNING

Risk of anaphylaxis.

Life-threatening anaphylactic reactions have been observed in some patients during ALDURAZYME infusions.  Therefore, appropriate medical support should be readily available when ALDURAZYME is administered.  Patients with compromised respiratory function or acute respiratory disease may be at risk of serious acute exacerbation of their respiratory compromise due to infusion reactions, and require additional monitoring.

 

Life-threatening anaphylactic reactions have been observed in some patients during or up to 3 hours after ALDURAZYME infusions. Reactions have included: respiratory failure, respiratory distress, stridor, tachypnea, bronchospasm, airway obstruction, hypoxia, hypotension, bradycardia, and urticaria. Interventions have included: resuscitation, mechanical ventilatory support, emergency tracheotomy, hospitalization, and treatment with inhaled beta-adrenergic agonists, epinephrine, and intravenous corticosteroids.

 

In clinical trials and postmarketing safety experience with ALDURAZYME, approximately 1% of patients experienced severe or serious allergic reactions. In patients with MPS I, pre-existing upper airway obstruction may have contributed to the severity of some reactions. Due to the potential for severe allergic reactions, appropriate medical support should be readily available when ALDURAZYME is administered. Because of the potential for recurrent reactions, some patients who experience initial severe reactions may require prolonged observation. The risks and benefits of re-administering ALDURAZYME following an anaphylactic or severe allergic reaction should be considered.

 

Patients with an acute illness at the time of ALDURAZYME infusion may be at greater risk for infusion-related reactions. Careful consideration should be given to the patient’s clinical status prior to administration of ALDURAZYME.

 

Patients should receive antipyretics and/or antihistamines prior to infusion. If an infusion reaction occurs, regardless of pretreatment, decreasing the infusion rate, temporarily stopping the infusion, and/or administration of additional antipyretics and/or antihistamines may ameliorate the symptoms.

 

The most common adverse reactions associated with ALDURAZYME treatment in the clinical studies were upper respiratory tract infection, rash, and injection site reaction  The most common adverse reactions requiring intervention were infusion-related reactions involving flushing, fever, headache, and rash. 

 

In postmarketing experience with ALDURAZYME, severe and serious infusion-related reactions have been reported, some of which were life-threatening.  The most frequently reported adverse reactions included: chills, vomiting, nausea, arthralgia, diarrhea, tachycardia, abdominal pain, blood pressure increased, and oxygen saturation decreased. 

 

Approximately 91% of patients treated with ALDURAZYME in clinical studies were positive for antibodies to laronidase. The clinical significance of antibodies to ALDURAZYME is not known, including the potential for product neutralization. Adverse events should be reported promptly to Genzyme Medical Information at 800-745-4447, option 2. ALDURAZYME is available by prescription only. To learn more, please see the full prescribing information (PDF) including boxed warning, visit www.ALDURAZYME.com or contact Genzyme at 1-800-745-4447.
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